(19)
(11) EP 4 438 115 A3

(12) EUROPEAN PATENT APPLICATION

(88) Date of publication A3:
08.01.2025 Bulletin 2025/02

(43) Date of publication A2:
02.10.2024 Bulletin 2024/40

(21) Application number: 24178429.7

(22) Date of filing: 04.09.2015
(51) International Patent Classification (IPC): 
A61K 48/00(2006.01)
C12N 9/22(2006.01)
C12N 15/85(2006.01)
A61P 7/06(2006.01)
C07K 14/805(2006.01)
C12N 15/113(2010.01)
C12N 15/867(2006.01)
(52) Cooperative Patent Classification (CPC):
C07K 14/805; C12N 9/22; C12N 15/86; C12N 2740/15043; C12N 2830/40; C12N 2740/16043; C12N 2830/15; C12N 2830/48; C12N 2830/50; A61K 48/0058; C12N 15/85; C12N 2830/008; C12N 2830/30; C12N 2830/46; A61K 48/0066; A61P 7/06
(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

(30) Priority: 04.09.2014 US 201462045997 P

(62) Application number of the earlier application in accordance with Art. 76 EPC:
19185730.9 / 3620521
15837740.8 / 3189143

(71) Applicants:
  • Memorial Sloan-Kettering Cancer Center
    New York, NY 10065 (US)
  • University of Washington
    Seattle, WA 98105-4721 (US)

(72) Inventors:
  • SADELAIN, Michel
    New York, 10024 (US)
  • RIVIERE, Isabelle
    New York, 10024 (US)
  • MANSILLA-SOTO, Jorge
    Forest Hills, 11375 (US)
  • WANG, Xiuyan
    New York, 10065 (US)
  • STAMATOYANNOPOULOS, George
    Seattle, 98105 (US)
  • STAMATOYANNOPOULOS, John
    Seattle, 98105 (US)
  • LIU, Mingdong
    Seattle (US)

(74) Representative: Barker Brettell LLP 
100 Hagley Road Edgbaston
Birmingham B16 8QQ
Birmingham B16 8QQ (GB)

   


(54) GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES


(57) The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.







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